Investigators report a novel approach for delivering large transgenes in lentiviruses, in which they demonstrated proof-of-concept for a ‘template-switching’ lentiviral vector that harnesses recombination events during reverse-transcription. During this work, they discovered that a standard, unmodified lentiviral vector was efficient in delivering full-length dystrophin to target cells, within a total genomic load of more than 15,000 base pairs. [Sci Rep]
Full Article

The authors sought to determine the safety and efficacy of allogeneic cardiosphere-derived cells (CDCs) in a large animal (canine) model of spontaneous dilated cardiomyopathy (DCM). Canine CDCs (cCDCs) were grown from a donor dog heart. Similar to human CDCs, cCDCs express CD105 and are slightly positive for c-kit and CD90. Thirty million of allogeneic cCDCs were infused into the coronary vessels of Doberman pinscher dogs with spontaneous DCM. [J Cell Mol Med]
Full Article

Researchers review several randomized trials of charged-particle therapies that are ongoing, with results that will enable selective delivery to patients who are most likely to benefit from them. They also discuss aspects related to radiobiology, including the immune response and hypoxia, which will need to be taken into consideration in future randomized trials to fully exploit the potential of charged particles. [Nat Rev Clin Oncol]
Abstract

The authors discuss the clinical outcome of current and emerging strategies of T-cell depletion in allogeneic hematopoietic stem cell transplant that have developed during the modern transplantation era, focusing specifically on ex vivo strategies that target selected T-cell subsets. [Bone Marrow Transplant]
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Audentes Therapeutics, Inc. announced that data from RECENSUS, a medical chart review of patients with XLMTM, will be presented. These data provide new insights into the significant medical burden for children with XLMTM, their families and caregivers. [Press release from Audentes Therapeutics, Inc. discussing research presented at the 2017 Muscular Dystrophy Association (MDA) Scientific Conference, Arlington]
Press Release

Medicenna BioPharma Inc. announced that it has entered into a sponsored research agreement with The University of Texas MD Anderson Cancer Center to develop next generation fusion protein therapeutics targeting the IL-4 Receptor. [Medicenna BioPharma Inc.]
Press Release

Ferring Pharmaceuticals and Brazil-based Instituto de Ciências Farmacêuticas (ICF) announced a research collaboration aimed at developing a mucoadhesive rectal delivery system to treat anal fissures and to improve quality of care for patients with inflammatory bowel disease (IBD). [Ferring Pharmaceuticals]
Press Release

Allergan plc and Editas Medicine, Inc. announced that Allergan’s wholly-owned subsidiary, Allergan Pharmaceuticals International Limited, and Editas Medicine have entered into a strategic research and development alliance under which Allergan will receive exclusive access and the option to license up to five of Editas Medicine’s genome-editing ocular programs, including its lead program for Leber Congenital Amaurosis, which is currently in pre-clinical development. [Allergan plc]
Press Release

Pharnext SA announced that the first two patients have entered the international Phase III Extension Study PLEO-CMT-FU of PXT3003 at La Timone University Hospital. PXT3003 is Pharnext’s lead PLEODRUG™ for the treatment of patients with mild-to-moderate Charcot-Marie-Tooth Disease Type 1A, a rare and debilitating inherited peripheral neuropathy for which there are no satisfactory approved treatments available. [Pharnext SA]
Press Release

Mesoblast Limited announced 36-month results from the randomized, placebo-controlled 100-patient Phase II trial of its proprietary allogeneic Mesenchymal Precursor Cells (MPCs) in patients with chronic low back pain due to intervertebral disc degeneration. A single intra-discal injection of 6 million MPCs resulted in meaningful improvements in both pain and function that were durable for at least 36 months. [Mesoblast Limited]
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NightstaRx Ltd. announces that the company has commenced enrolling and dosing subjects in a Phase I/II clinical trial to treat patients with X-Linked Retinitis Pigmentosa (XLRP). This is the first clinical trial of its type in XLRP worldwide, and has been progressed from licensing to clinical trial in a period of just 18 months. [NightstaRx Ltd.]
Press Release

RepliCel Life Sciences Inc. announced the successful completion of its first-in-human clinical study of the company’s autologous cell therapy for the treatment of androgenetic alopecia (pattern baldness). [RepliCel Life Sciences Inc.]
Press Release

Longeveron announced the successful completion of enrollment in the first phase of its clinical research trial of Longeveron allogeneic human mesenchymal stem cells to treat Alzheimer’s disease. [Longeveron (PR Newswire Association LLC.)]
Press Release

Fate Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug application for FATE-NK100, the company’s first-in-class adaptive memory natural killer cell product candidate. The company expects a first-in-human clinical trial of FATE-NK100 for advanced acute myeloid leukemia to open enrollment at the Masonic Cancer Center, University of Minnesota following approval of the center’s institutional review board. [Fate Therapeutics, Inc.]
Press Release

Kadimastem announced that it has been granted a patent from the United States Patent and Trademark Office for its technology in the field of cell based treatment for diseases of the nervous system. The United States patent joins additional patents granted in Israel and Europe. [Kadimastem]
Press Release

Arbutus Biopharma Corporation announced it has licensed to Alexion Pharmaceuticals, Inc. its proprietary lipid nanoparticle (LNP) technology for exclusive use in one of Alexion’s rare disease programs. This transaction enables Alexion to address delivery for therapeutic application of messenger RNA (mRNA) and rapidly enter clinical development with its mRNA product candidate. [Arbutus Biopharma Corporation]
Press Release

Stem cell science belongs to Canada and it is powering regenerative medicine. Leaders from across this emerging sector have joined forces to advance the field, through the newly created Regenerative Medicine Alliance of Canada. [University of Toronto]
Press Release

An Iranian researcher jailed in Tehran for the last 11 months is in declining health after spending more than two months on hunger strike. This month, researchers around the world made urgent appeals for his release. [Nature News]
Editorial

The Trump administration may be planning to help pay for a massive 18% cut to the National Institutes of Health (NIH) by slashing payments to universities and research institutes for overhead costs, ScienceInsider has learned. [ScienceInsider]
Editorial